Key FDA Approvals Include First OTC, Nonprescription Daily Oral Contraceptive, First Monoclonal Injection to Prevent RSV in Infants, and More
FDA Approves First Over-The-Counter, Nonprescription Daily Oral Contraceptive1
The FDA has approved the first nonprescription, progestin-only daily oral contraceptive—norgestrel—for the prevention of pregnancy.
“When used as directed, daily oral contraception is safe and is expected to be more effective than currently available nonprescription contraceptive methods in preventing unintended pregnancy,” Patrizia Cavazzoni, MD, director of the FDA’s Center for Drug Evaluation and Research, said in a press release.
Studies were able to establish that consumers had a high understanding of information on the product’s Drug Facts label. Further, a high proportion of consumers understood the label instructions, which supported their ability to use norgestrel.
The switch from prescription use to nonprescription use required the applicant to show that the “product can be used by consumers safely and effectively, relying only on the nonprescription drug labeling without any assistance from a health care professional.”
FDA Approves First Monoclonal Injection to Prevent RSV in Infants, Toddlers2
The FDA has approved the first monoclonal injection for neonates, infants, and toddlers for the prevention of syncytial virus (RSV). Nirsevimab-alip—a monoclonal antibody—was approved for use in neonates and infants born during or entering their first RSV season and in children aged up to 24 months who are vulnerable to severe RSV disease through their second season of the disease.
Monoclonal antibodies are laboratory-made proteins that are meant to mimic the immune system’s ability to fight off viruses. A singular, intramuscular dose of nirsevimab-alip prior to or during the RSV system may offer protection during the virus’s season.
“RSV can cause serious disease in infants and some children and results in a large number of emergency department and physician office visits each year,” John Farley, MD, MPH, director of the Office of Infectious Diseases in the FDA’s Center for Drug Evaluation and Research, said in a press release. “Today’s approval addresses the great need for products to help reduce the impact of RSV disease on children, families and the health care system.”
FDA Converts Alzheimer Disease Treatment to Traditional Approval3
Following the results of a recent confirmatory clinical trial, the FDA converted the use of lecanemab-irmb for the treatment of adult patients with Alzheimer disease to a traditional approval. It was initially approved for use in January 2023 under the accelerated approval pathway.
Lecanemab reduces amyloid plaques, which is a key pathophysiological aspect of Alzheimer disease in the brain. This approval is the first amyloid beta-directed antibody to be converted to a traditional approval for Alzheimer disease treatment.
The Study 301 (CLARITY AD) phase 3 randomized controlled trial was conducted to verify clinical benefit of lecanemab. The researchers included a total of 1795 adults with Alzheimer disease and either mild cognitive impairment or mild dementia stage of disease, as well as confirmed amyloid beta pathology. Patients were randomized to receive lecanemab 10mg/kg once every two weeks or placebo. When compared with placebo, the treatment group had statistically significant and clinically meaningful reduction of decline from baseline to 18 months on the Clinical Dementia Rating Scale Sum of Boxes score, which was the primary endpoint. Similar results were observed for the secondary endpoints, including the Alzheimer Disease Assessment Scale Cognitive Subscale 14 and the Alzheimer Disease Cooperative Study—Activities of Daily Living Scale for Mild Cognitive Impairment.
First Treatment for Pediatric Functional Constipation Approved by FDA4
The FDA has approved linaclotide to treat functional constipation in pediatric patients aged 6 to 17 years of age—the first treatment of its kind for this population.
A common condition, functional constipation is experienced by children and adolescents. Patients experience infrequent bowel movements with hard stools that can be difficult and painful to pass. Currently, there is no known underlying organic cause, and there are typically multiple contributing factors, according to the press release.
The approval follows a 12-week double-blind, placebo-controlled, randomized, multicenter clinical trial. Additionally, the trial was supported by efficacy data from “adequate and well-controlled trials in adults with chronic idiopathic constipation (constipation that persists and isn’t connected to an underlying illness),” according to the press release.
Topical Treatment for Molluscum Contagiosum in Adults, Children Approved by FDA5
The FDA has approved the first treatment for molluscum contagiosum in adults and pediatric patients aged 2 years and older. Cantharidin is a topical treatment that can be used on the skin and is administered to patients only by their health care providers.
Molluscum contagiosum is a viral skin infection that causes white, pink, or flesh-colored bumps to appear on the skin that may itch or become irritated. The bumps can happen alone or in groups anywhere on a person’s skin.
The approval of cantharidin follows two double-blind, randomized, placebo-controlled trials that tested the efficacy of treatment. In total, 528 participants aged 2 years to 60 years old with molluscum contagiosum were randomized to receive either treatment with cantharidin or placebo. Participants received treatment at 21-day intervals until bumps on the skin were completely cleared or for a maximum of four applications. Overall, 54% of subjects treated with cantharidin achieved complete clearance of all treated molluscum bumps by day 84—the primary efficacy endpoint. This result was compared to the 13% of subjects treated with placebo that achieved the same endpoint.
FDA Approves First Pancreatic Islet Cell Therapy for Adults with Type 1 Diabetes6
The FDA has approved the first pancreatic islet cellular therapy for the treatment of type 1 diabetes. The treatment is made from deceased donor pancreatic cells and is intended to treat adults with type 1 diabetes who, despite extensive diabetes management, cannot reach average blood glucose levels because of repeated episodes of severe low blood sugar.
“Severe hypoglycemia is a dangerous condition that can lead to injuries resulting from loss of consciousness or seizures,” Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research said in a press release. “Today’s approval, the first-ever cell therapy to treat patients with type 1 diabetes, provides individuals living with type 1 diabetes and recurrent severe hypoglycemia an additional treatment option to help achieve target blood glucose levels.”
Treatment is administered as a single infusion into the hepatic portal vein with an additional infusion being performed depending on the patient’s response to the first dose. According to the FDA’s press release, the “primary mechanism of action of [the therapy] is believed to be the secretion of insulin by the infused allogeneic islet beta cells.”
References:
- FDA approves first nonprescription daily oral contraceptive. News release. The US Food and Drug Administration; July 13, 2023. Accessed July 21, 2023. www.fda.gov/news-events/press-announcements/fda-approves-first-nonprescription-daily-oral-contraceptive.
- FDA approves new drug to prevent RSV in babies and toddlers. News release. US Food and Drug Administration; July 17, 2023. Accessed July 24, 2023. www.fda.gov/news-events/press-announcements/fda-approves-new-drug-prevent-rsv-babies-and-toddlers.
- FDA converts novel alzheimer’s disease treatment to traditional approval. News release. The US Food and Drug Administration; July 6, 2023. Accessed July 24, 2023. https://www.fda.gov/news-events/press-announcements/fda-converts-novel-alzheimers-disease-treatment-traditional-approval.
- FDA approves first treatment for pediatric functional constipation. News release. US Food and Drug Administration; June 12, 2023. Accessed June 14, 2023. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-first-treatment-pediatric-functional-constipation.
- FDA approves first treatment for molluscum contagiosum. News release. US Food and Drug Administration; July 24, 2023. Accessed July 26, 2023. www.fda.gov/drugs/news-events-human-drugs/fda-approves-first-treatment-molluscum-contagiosum
- FDA approves first cellular therapy to treat patients with type 1 diabetes. News release. US Food and Drug Administration; June 28, 2023. Accessed July 18, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-first-cellular-therapy-treat-patients-type-1-diabetes